Myelodysplastic Syndromes (MDS)

Canakinumab or emavusertib for anemia in lower-risk MDS: results of the CANFIRE and LUCAS phase 2, open-label, multicenter trials

Background: Dysregulated innate immune signaling promotes inflammatory suppression of hematopoietic stem and progenitor cell function, contributing to ineffective erythropoiesis in myelodysplastic neoplasms (MDS). The phase 2, open-label CANFIRE and LUCAS trials aimed to determine the efficacy and safety of canakinumab or emavusertib, respectively, in patients with lower-risk MDS (LR-MDS) or myelodysplastic/myeloproliferative neoplasms (MDS/MPN).

Immunotherapeutic targeting of calreticulin mutant myeloproliferative neoplasms

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Abstract Calreticulin (CALR) frameshift mutations drive the majority of JAK2/MPL-wild-type cases of essential thrombocythemia and myelofibrosis, producing a shared novel C-terminus that activates the thrombopoietin receptor and leads to constitutive Janus kinase (JAK)/signal…

Post-transplant hypomethylating agents plus venetoclax maintenance improves survival in high-risk myeloid malignancies: A matched cohort study

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Relapse is the leading cause of allogeneic hematopoietic stem cell transplant (allo-HSCT) failure in myeloid neoplasms [1]. Patients receiving reduced-intensity conditioning (RIC) regimens have a higher risk of post-transplant relapse compared to those undergoing myeloablative…

Stag2-dependent chromatin remodeling enforces the erythroid-specific Gata1 cistrome

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Key Points Stag2 reprograms Gata1 cistrome specificity via chromatin accessibility, a key determinant of transcription factor lineage discrimination. Loss of the Stag2 rheostat causes accessibility-driven Gata1 retargeting, providing a mechanistic framework for dyserythropoiesis…

TA-TMA and GVHD are distinct consequences of endothelial injury: a MIDAS consortium study

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Key Points Posttransplant cyclophosphamide significantly reduced GVHD incidence but not TA-TMA incidence, which predated acute GVHD in ∼50% of cases. TA-TMA biomarker soluble C5b-9 was not significant in adults; pre-HCT serum creatinine, placental growth factor, and day +28 ST2…
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